近日，一支中國的科學(xué)家團(tuán)隊首次利用CRISPR-Cas9基因編輯技術(shù)，將編輯過的細(xì)胞注入了一名病人體內(nèi)。這也是該技術(shù)在全球首次被直接應(yīng)用于人體，在基因研究領(lǐng)域具有里程碑式的意義。

據(jù)《自然》雜志報道，10月28日，四川大學(xué)的研究人員們在四川大學(xué)華西醫(yī)院將經(jīng)過基因編輯的細(xì)胞注入到了一名參與臨床試驗的肺癌患者體內(nèi)。這支團(tuán)隊是由腫瘤學(xué)家盧鈾醫(yī)生領(lǐng)導(dǎo)的。

這種旨在治療癌癥的CRISPR基因編輯技術(shù)需要進(jìn)行一系列的分子切割，引導(dǎo)Cas9分子“敲”掉免疫細(xì)胞內(nèi)可能滋生癌癥的致病基因。然后這些被編輯過的細(xì)胞會被重新放回患者體內(nèi)，用以攻擊惡性腫瘤。

此次人體試驗原本計劃于今年八月進(jìn)行，由于培養(yǎng)被編輯的細(xì)胞所需要的時間超出了最初的預(yù)期，因而一直拖到十月底才得以實施。不過中國人在CRISPR技術(shù)上的試驗步伐依然遠(yuǎn)遠(yuǎn)走在了美國人的前頭。美國的首次CRISPR人體試驗?zāi)壳斑€處于計劃階段，雖然今年年初，美國的研究人員已經(jīng)獲準(zhǔn)進(jìn)行此項試驗（并且獲得了科技界的億萬富翁肖恩·帕克的投資），但估計一直要等到2017年年初，這項試驗才會在賓西法尼亞大學(xué)進(jìn)行。

癌癥免疫學(xué)家卡爾·瓊恩博士對《自然》雜志表示，中國科學(xué)家率先實施此項技術(shù)的臨床測試，很可能會引發(fā)中美兩國在此項技術(shù)上的良性競爭。

“我認(rèn)為在生物醫(yī)學(xué)領(lǐng)域上將爆發(fā)一場新的‘美蘇太空競賽’，不過這次的競爭雙方將是中國和美國。不過競爭也是很重要的，因為它通常有助于提高最終產(chǎn)品的質(zhì)量。”

中國科學(xué)家的此次試驗將在10名患者身上進(jìn)行，被試者每人將接受2至4次的編輯細(xì)胞注射。該研究的重點在于確定這項基因療法的安全性，以及它是否會產(chǎn)生令人無法接受的副作用。（財富中文網(wǎng)）

譯者：樸成奎

In a landmark for genomic research, a team of Chinese scientists has injected cells modified with the groundbreaking CRISPR-Cas9 gene-editing technology into a patient. It’s the first known time the technique has actually been deployed in a human.

Researchers from the Sichuan University in Chengdu inserted the re-engineered cells into a lung cancer patient participating in a clinical trial at the West China Hospital on October 28th, according to Nature. The team is being led by oncologist Dr. Lu You.

Cancer-focused CRISPR technology involves taking a set of molecular shears and the guiding molecule Cas9 in order to cut out unwanted genes in immune cells that may help proliferate cancers. These modified cells are then put back into patients in order to attack cancerous tumors.

The trial was actually supposed to launch back in August but was delayed because growing and culturing the genomically edited cells took longer than originally expected. Regardless, the study has begun well ahead of the first planned American CRISPR trials, which won regulatory clearance earlier this year (and are being funded by tech billionaire Sean Parker) but are unlikely to begin until 2017 at the University of Pennsylvania.

Still, the Chinese scientists’ first-to-the-clinic victory may help fuel positive rivalries in the U.S., scientist and cancer immunotherapy expert Dr. Carl June told Nature.

“I think this is going to trigger ‘Sputnik 2.0’, a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product,” he said.

The Chinese trial will be conducted on ten patients who will receive anywhere from two to four injections of modified cells, and the study will mostly focus on how safe the treatments are and whether or not they have unacceptable side effects.